The U.K. National Institute for Health and Care Excellence recommended Pfizer Inc.'s blood cancer drug Besponsa in a final draft guidance after the company provided additional information and agreed to a confidential discounted price.
The U.K. regulator had rejected Besponsa in August 2017 over uncertainties regarding its survival benefits and cost-effectiveness.
In the final guidance, the regulator approved Besponsa, or inotuzumab ozogamicin, for treating adults with an aggressive, rare form of blood cancer called CD22-positive B cell precursor acute lymphoblastic leukemia.
The regulator said it recommended the therapy after Pfizer provided further evidence of the drug's effectiveness and agreed to a confidential discounted price.
NICE said Besponsa reduces the need for hospitalization and enables more people to be eligible for stem cell transplants compared to other treatments. Patients who have philadelphia-chromosome-positive acute lymphoblastic leukemia must have failed treatment with a tyrosine kinase inhibitor first before they can be offered Besponsa, the watchdog added.
About 650 people in the U.K. are diagnosed with acute lymphoblastic leukemia annually, and around 114 of these patients will be eligible for treatment with Besponsa.
Besponsa is approved by the U.S. Food and Drug Administration for the same indication.